AbelZeta's C-CAR168 Cleared by FDA for Phase 2 Registrational Trial in Refractory Lupus Nephritis

AbelZeta Pharma has received FDA clearance, under the agency's regenerative medicine advanced therapy (RMAT) designation, for a multiple-center registrational phase 2 trial of C-CAR168 in patients with lupus nephritis (LN) refractory to standard therapy.¹ C-CAR168 is an anti-CD20/b-cell maturation antigen (BCMA) bispecific chimeric antigen receptor T cell (CAR-T) therapy.¹

"The FDA clearance to proceed with this multiple-center registrational [phase 2] trial for C-CAR168 in LN represents an important step forward in the development of this promising new modality for patients with severe, treatment-refractory lupus," said Tony (Bizuo) Liu, chairman and chief executive officer of AbelZeta, in a company press release.¹

What will the phase 2 trial evaluate?

The registrational trial will further assess the safety and efficacy of C-CAR168 in patients with LN who have not responded to standard treatment, a population the company described as having significant unmet medical need. The study will be conducted across multiple clinical centers.

Liu noted in the release that C-CAR168 has also received priority medicines (PRIME) designation from the European Medicines Agency (EMA) and said the combination of RMAT and PRIME status "underscores the tremendous potential of C-CAR168 to address a significant unmet need for autoimmune diseases."¹ He added that the company is actively evaluating the potential of C-CAR168 in other major disease indications.¹

What is RMAT designation?

RMAT designation is FDA’s expedited program, established under the 21st Century Cures Act, for regenerative medicine therapies, including cell therapies, tissue engineering products, and certain combination products. The designation applies to such medicines that are intended to treat, modify, reverse, or cure a serious or life-threatening disease, where preliminary clinical evidence suggests the therapy could address an unmet medical need.²

RMAT designation offers benefits similar to breakthrough therapy status, including more frequent FDA interactions and potential eligibility for accelerated approval and priority review.² PRIME, the EMA's parallel program, similarly offers early and proactive regulatory support, including a dedicated rapporteur and eligibility for accelerated assessment, for medicines targeting conditions with significant unmet medical need.³

What is C-CAR168 designed to do?

C-CAR168 is a novel autologous bispecific CAR-T therapy that targets both CD20 and BCMA. It is designed to deplete disease-driving B cells and plasma cells, with the goal of inducing an immune reset in patients with severe autoimmune diseases, including refractory systemic lupus erythematosus (SLE) and LN, as well as progressive multiple sclerosis (MS).¹ FDA and EMA have granted RMAT and PRIME designations, respectively, to C-CAR168 for the treatment of refractory SLE, including LN.¹

Why does refractory LN remain a treatment challenge?

SLE is a chronic autoimmune disease marked by systemic inflammation and involvement of multiple organs, and LN—a serious manifestation of SLE involving kidney inflammation—can progress to kidney damage and kidney failure.¹ Despite existing standards of care, patients with refractory disease continue to face persistent disease activity, risk of organ damage, and cumulative toxicity from long-term immunosuppressive treatment, underscoring the need for new treatment approaches such as cell-based immunotherapies.¹

What’s next for AbelZeta?

AbelZeta, a US and China-based clinical-stage biopharmaceutical company, has centers of excellence in Rockville, Md., and Shanghai, China. The company focuses on developing proprietary cell-based therapeutics for hematological malignancies, inflammatory and immunological diseases, and solid tumors.

The company aims to develop bespoke treatments that harness the body's own immune system to fight disease. It conducts R&D activities at its own good manufacturing practice facilities at its centers of excellence with the goal of maintaining a pipeline comprised of multiple CAR-T therapies.¹

References

1. AbelZeta Pharma. AbelZeta receives FDA clearance of registrational Phase II trial for C-CAR168 in refractory lupus nephritis. PR Newswire. Published July 27, 2026. Accessed July 28, 2026. https://prnmedia.prnewswire.com/news-releases/abelzeta-receives-fda-clearance-of-registrational-phase-ii-trial-for-c-car168-in-refractory-lupus-nephritis-302835248.html
2. FDA. Regenerative medicine advanced therapy designation. Center for Biologics Evaluation and Research. FDA.gov. Updated November 4, 2025. Accessed July 28, 2026. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation
3. European Medicines Agency. PRIME: priority medicines. EMA.europa.eu Accessed July 28, 2026. https://www.ema.europa.eu/en/human-regulatory-overview/research-development/prime-priority-medicines